Clinical trials
Not yet recruiting Genetic Diseases, Inborn trials
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
14 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2024 | 1 |
| 2025 | 2 |
| 2026 | 8 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Not yet recruiting | 14 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| Assistance Publique - Hôpitaux de Paris | 2 |
| Adjuvia Therapeutics, Inc. | 1 |
| Agnieszka Czechowicz | 1 |
| Genethon | 1 |
| Hospices Civils de Lyon | 1 |
| Izmir Ataturk Training and Research Hospital | 1 |
| Johns Hopkins University | 1 |
| NeuroGT | 1 |
| Rocket Pharmaceuticals Inc. | 1 |
| The Hospital for Sick Children | 1 |
Top conditions
MeSH conditions the trials were registered with, not their broader categories. A condition with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| Genetic Diseases, Inborn | 14 |
| Cardiomyopathies | 2 |
| Central Nervous System Diseases | 2 |
| Congenital, Hereditary, and Neonatal Diseases and Abnormalities | 2 |
| Metabolic Diseases | 2 |
| Metabolism, Inborn Errors | 2 |
| Rare Diseases | 2 |
| Amino Acid Metabolism, Inborn Errors | 1 |
| Anemia, Hypoplastic, Congenital | 1 |
| Ataxia | 1 |
Trials
Showing 26 to 14 of 14. Each trial links to its page on Pharma Observatory.
No trials match these filters.
Try a different keyword, or clear a filter.
Recently changed trials
Changes observed in the last 7 days, newest first, site-count changes left out. Source: ClinicalTrials.gov.
No changes to these trials in the last 7 days.
Go deeper on Pharma Observatory
Pharma Observatory connects clinical trials, sponsors, sites and investigators in one searchable platform.